Cell and Gene Therapy Today—April 2, 2026

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🧬 Rocket wins first US approval in severe LAD-I [1] [US • 27 Mar 2026]

https://www.businesswire.com/news/home/20260326279809/en/Rocket-Pharmaceuticals-Announces-FDA-Approval-of-KRESLADI-for-Pediatric-Patients-with-Severe-Leukocyte-Adhesion-Deficiency-I-LAD-I
Context: The approval is based on increased neutrophil CD18 and CD11a surface expression, with confirmation of clinical benefit to come from longer-term follow-up and a post-marketing registry [1].
Key point: KRESLADI (Rocket Pharmaceuticals; marnetegragene autotemcel) received FDA accelerated approval for pediatric patients with severe leukocyte adhesion deficiency-I due to biallelic ITGB2 variants, without an available HLA-matched sibling donor for allogeneic HSCT [1].
Implication: May influence prescriber choice and payer reviews pending full data.

🧠 Encoded starts pivotal path for ETX101 in Dravet syndrome [2] [US • 25 Mar 2026]

https://encoded.com/press-releases/encoded-therapeutics-announces-fda-alignment-initiation-of-pivotal-study-and-asgct-presidential-symposium-presentation-of-etx101-in-dravet-syndrome/
Context: ENDEAVOR Part 2 is a randomized, double-blind, sham delayed-treatment controlled study in 30 children aged 6 months to under 4 years over 52 weeks; ENDEAVOR Part 1B is expanding into older patients aged 4 years to under 18 years [2].
Key point: Encoded Therapeutics said FDA aligned on the pivotal design for ETX101, an AAV9-based gene regulation therapy for SCN1A+ Dravet syndrome, supporting a planned BLA path [2].
Implication: May influence prescriber choice and payer reviews pending full data.

🧪 Beam picks 60 mg for BEAM-302 pivotal development [3] [US • 25 Mar 2026]

https://investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-announces-compelling-updated-clinical-data
Context: In the 60 mg cohort, Beam reported mean steady-state total AAT of 16.1 µM, corrected M-AAT comprising 94% of total AAT, and an 84% reduction in mutant Z-AAT, with follow-up up to 12 months; the company described the single-dose safety profile as well tolerated up to 75 mg [3].
Key point: Beam Therapeutics reported updated Phase 1/2 data for BEAM-302 in alpha-1 antitrypsin deficiency and selected 60 mg as the optimal biological dose for pivotal development [3].
Implication: May influence prescriber choice and payer reviews pending full data.

💧 MeiraGTx gains Breakthrough status in radiation-induced xerostomia [4] [UK/US • 26 Mar 2026]

https://investors.meiragtx.com/news-releases/news-release-details/meiragtx-announces-fda-breakthrough-therapy-designation-aav2
Context: The company said the designation was supported by 3-year Phase 1 dose-escalation data, and that FDA had already granted RMAT designation; MeiraGTx also said AQUAx2 is aligned with FDA for a potential BLA-supporting path [4].
Key point: MeiraGTx announced FDA Breakthrough Therapy Designation for AAV2-hAQP1 in Grade 2 and Grade 3 radiation-induced xerostomia after radiotherapy for upper aerodigestive tract cancers [4].
Implication: May influence prescriber choice and payer reviews pending full data.

🧠 AviadoBio moves AVB-101 into a fourth cohort in FTD-GRN [5] [UK • 31 Mar 2026]

https://www.businesswire.com/news/home/20260330432799/en/AviadoBio-Advances-AVB-101-Gene-Therapy-Program-and-Initiates-Fourth-Dose-Escalation-Cohort-of-Phase-12-ASPIRE-FTD-Trial
Context: The company said 12 patients across five countries had been dosed in the first three cohorts, early data showed dose-dependent CSF progranulin increases, and the overall safety profile remained favorable with no serious adverse events related to AVB-101 reported to date [5].
Key point: AviadoBio initiated enrollment in the fourth dose-escalation cohort of the Phase 1/2 ASPIRE-FTD trial for AVB-101 in frontotemporal dementia with GRN mutations [5].
Implication: May influence prescriber choice and payer reviews pending full data.

👁️ VeonGen reports early functional signals for VG801 in Stargardt disease [6] [EU • 25 Mar 2026]

https://www.globenewswire.com/news-release/2026/03/25/3262229/0/en/VeonGen-Announces-Clinical-Progress-of-VG801-Gene-Therapy-in-Stargardt-Disease-with-an-Oral-Presentation-at-ARVO-2026.html
Context: The company reported consistent improvements in BCVA and Virtual Reality Visual Test, with nine patients completing six-month follow-up and some reaching 12 months; no dose-limiting or serious adverse events were reported to date [6].
Key point: VeonGen said VG801 showed encouraging preliminary efficacy in an ongoing Phase 1/2 study in ABCA4-associated retinal disease, including Stargardt disease [6].
Implication: May influence prescriber choice and payer reviews pending full data.

🩸 AstraZeneca’s in vivo CAR-T shows early activity, but safety remains central [7] [China • 27 Mar 2026]

https://www.fiercebiotech.com/biotech/astrazenecas-vivo-car-t-bet-eradicates-cancer-3-5-patients-death-mars-dataset
Context: The same report said all patients had Grade 3 or higher adverse events, cytokine release syndrome occurred in four patients, and one patient died after spinal cord compression related to a lesion; the authors said anatomical risk stratification is important in patients with preexisting extramedullary disease [7].
Key point: A Fierce Biotech report on a Nature Medicine paper said EsoBiotec’s in vivo BCMA CAR-T candidate ESO-T01 showed objective responses in four of five patients with multiple myeloma, including three stringent complete remissions by day 60 [7].
Implication: May influence prescriber choice and payer reviews pending full data.

🧬 Caribou secures RMAT for off-the-shelf BCMA CAR-T [8] [US • 31 Mar 2026]

https://www.bitget.com/amp/news/detail/12560605321733
Context: The announcement cited previously disclosed CaMMouflage Phase 1 dose-escalation data in 12 BCMA-naïve patients at the recommended dose for expansion, including 92% ORR, 75% at or above complete response, and 91% MRD negativity in evaluable patients; the company also described a manageable safety profile (follow-up and cohort context per source) [8].
Key point: Caribou Biosciences said FDA granted RMAT designation to CB-011, an allogeneic anti-BCMA CAR-T cell therapy, for relapsed or refractory multiple myeloma [8].
Implication: May influence prescriber choice and payer reviews pending full data.

Why It Matters

FDA and RMAT-related milestones were a clear theme this week, spanning Rocket, Encoded, MeiraGTx, VeonGen, and Caribou [1][2][4][6][8].
Rare disease gene therapy remained active across immunology, neurology, ophthalmology, and salivary gland repair, showing how broad the modality footprint has become [1][2][4][5][6].
Several programs moved from early clinical signal toward registrational planning, especially ETX101 and BEAM-302 [2][3].
Biomarker-led development remained important, including neutrophil surface expression in LAD-I, AAT biomarkers in AATD, CSF progranulin in FTD-GRN, and functional vision endpoints in Stargardt disease [1][3][5][6].
In myeloma, the contrast was notable: in vivo CAR-T feasibility and response signals were paired with meaningful safety concerns, while an allogeneic anti-BCMA CAR-T advanced through FDA’s RMAT pathway [7][8].

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FAQ

What exactly was approved for Rocket’s KRESLADI?

KRESLADI (marnetegragene autotemcel) was granted FDA accelerated approval for pediatric patients with severe LAD-I due to biallelic ITGB2 variants who lack an available HLA-matched sibling donor for allogeneic HSCT [1]. The approval is tied to a surrogate marker, with longer-term confirmation still required [1].

What did FDA alignment mean for Encoded’s ETX101 program?

Encoded said FDA aligned on the pivotal study design for ETX101 in SCN1A+ Dravet syndrome, which supports the company’s planned BLA path [2]. The registrational study includes seizure reduction as the primary endpoint and cognition as a key secondary endpoint [2].

Why did Beam’s BEAM-302 update stand out?

Beam paired a dose-selection decision with updated biomarker data, choosing 60 mg for pivotal development in AATD [3]. The company reported total AAT above the 11 µM protective threshold in that cohort, along with high corrected M-AAT composition and reduced mutant Z-AAT [3].

What is notable about MeiraGTx’s AAV2-hAQP1 milestone?

The program now has both RMAT and Breakthrough Therapy Designation from FDA for Grade 2 and Grade 3 radiation-induced xerostomia [4]. That combination can support closer regulatory interaction as the AQUAx2 study progresses [4].

What should readers take from the AstraZeneca / EsoBiotec ESO-T01 report?

The report suggested early anti-myeloma activity from an in vivo CAR-T approach, but also highlighted substantial toxicity in a very small dataset, including one death [7]. The source frames the results as proof of feasibility, not clear differentiation from established CAR-T profiles [7].

Why does Caribou’s CB-011 RMAT matter?

RMAT can speed FDA interaction and review options for serious conditions with unmet need [8]. For CB-011, the designation adds regulatory momentum to an off-the-shelf anti-BCMA CAR-T program in relapsed or refractory multiple myeloma [8].

Entities / Keywords

Rocket Pharmaceuticals, KRESLADI, marnetegragene autotemcel, LAD-I, leukocyte adhesion deficiency-I, ITGB2
Encoded Therapeutics, ETX101, Dravet syndrome, SCN1A+, ENDEAVOR, POLARIS
Beam Therapeutics, BEAM-302, alpha-1 antitrypsin deficiency, AATD, SERPINA1, PiZ, base editing
MeiraGTx, AAV2-hAQP1, radiation-induced xerostomia, RIX, AQUAx2, AAV-GAD
AviadoBio, AVB-101, FTD-GRN, frontotemporal dementia, progranulin, ASPIRE-FTD
VeonGen, VG801, Stargardt disease, ABCA4-associated retinal disease, BCVA, VRVT
AstraZeneca, EsoBiotec, ESO-T01, in vivo CAR-T, BCMA, multiple myeloma
Caribou Biosciences, CB-011, allogeneic CAR-T, anti-BCMA, CaMMouflage, RMAT